For help on how to get the results you want, see our search tips.
140 results
-
List item
Direct healthcare professional communication (DHPC): Respreeza (human alpha-1-proteinase inhibitor): Batch-specific product recall
Active substance: alpha1-proteinase inhibitor (human), DHPC type: Quality defect, Last updated: 12/02/2021 -
List item
Direct healthcare professional communication (DHPC): Lojuxta (lomitapide): Reminder to monitor the liver function of patients treated with Lojuxta and to avoid use in pregnancy
Active substance: Lomitapide, DHPC type: Post-authorisation measure, Last updated: 17/02/2021 -
List item
Direct healthcare professional communication (DHPC): Strimvelis® (autologous CD34+ enriched cell fraction that contains CD34+ cells transduced with retroviral vector that encodes for the human adenosine deaminase [ADA] cDNA sequence): first case of lymphoid T cell leukaemia after insertional oncogenesis
Active substance: autologous CD34+ enriched cell fraction that contains CD34+ cells transduced with retroviral vector that encodes for the human adenosine deaminase (ADA) cDNA sequence from human haematopoietic stem/progenitor (CD34+) cells, DHPC type: Adverse event, Last updated: 22/03/2021 -
List item
Direct healthcare professional communication (DHPC): Xeljanz (tofacitinib): Initial clinical trial results of increased risk of major adverse cardiovascular events and malignancies (excluding NMSC) with use of tofacitinib relative to TNF— alpha inhibitors (new)
Active substance: tofacitinib citrate, DHPC type: Safety signal, Last updated: 24/03/2021 -
List item
Direct healthcare professional communication (DHPC): Respreeza (human alpha-1-proteinase inhibitor): Sterility issue with the infusion device co-packed with Respreeza 4.000 mg and 5.000 mg (new)
Active substance: Human alpha1-proteinase inhibitor, DHPC type: Medicine shortage, Last updated: 26/03/2021 -
List item
Direct healthcare professional communication (DHPC): Zolgensma (onasemnogene abeparvovec): risk for thrombotic microangiopathy
Active substance: onasemnogene abeparvovec, DHPC type: Type II variation, Last updated: 18/03/2021 -
List item
Direct healthcare professional communication (DHPC): Vaxzevria (previously COVID-19 Vaccine AstraZeneca): Risk of thrombocytopenia and coagulation disorders (new)
Active substance: Chimpanzee Adenovirus encoding the SARS CoV 2 Spike glycoprotein (ChAdOx1-S), DHPC type: Safety signal, Last updated: 24/03/2021 -
List item
Direct healthcare professional communication (DHPC): Vaxzevria (previously COVID-19 Vaccine AstraZeneca): link between the vaccine and the occurrence of thrombosis in combination with thrombocytopenia (new)
Active substance: ChAdOx1-SARS-COV-2, DHPC type: Safety signal, Last updated: 13/04/2021 -
List item
Orphan designation: Murine anti-CD22 antibody variable region fused to truncated Pseudomonas exotoxin 38 ( Moxetumomab pasudotox) for: Treatment of hairy cell leukaemia
Date of first decision: 05/12/2008, Positive, Last updated: 09/03/2021 -
List item
Orphan designation: N-tert-butyl-3-[(5-methyl-2-{[4-(2-pyrrolidin-1-ylethoxy)phenyl]amino}pyrimidin-4-yl)amino] benzenesulfonamide dihydrochloride monohydrate (fedratinib) for: Treatment of post-polycythaemia vera myelofibrosis
Date of first decision: 26/11/2010, Positive, Last updated: 03/03/2021 -
List item
Orphan designation: N-tert-butyl-3-[(5-methyl-2-{[4-(2-pyrrolidin-1-ylethoxy)phenyl]amino}pyrimidin-4-yl)amino] benzenesulfonamide dihydrochloride monohydrate (fedratinib) for: Treatment of post-essential thrombocythaemia myelofibrosis
Date of first decision: 26/11/2010, Positive, Last updated: 03/03/2021 -
List item
Orphan designation: N-tert-butyl-3-[(5-methyl-2-{[4-(2-pyrrolidin-1-ylethoxy)phenyl]amino}pyrimidin-4-yl)amino] benzenesulfonamide dihydrochloride monohydrate (fedratinib) for: Treatment of primary myelofibrosis
Date of first decision: 01/10/2010, Positive, Last updated: 03/03/2021 -
List item
Orphan designation: Recombinant fusion protein consisting of a modified form of the extracellular domain of human activin receptor IIB linked to the human IgG1 Fc domain (luspatercept) for: Treatment of beta thalassaemia intermedia and major
Date of first decision: 29/07/2014, Positive, Last updated: 18/02/2021 -
List item
Orphan designation: Recombinant fusion protein consisting of a modified form of the extracellular domain of human activin receptor IIB linked to the human IgG1 Fc domain (luspatercept) for: Treatment of myelodysplastic syndromes
Date of first decision: 22/08/2014, Positive, Last updated: 18/02/2021 -
List item
Orphan designation: Autologous peripheral blood T cells CD4 and CD8 selected and CD3 and CD28 activated transduced with retroviral vector expressing anti CD19 CD28/CD3-zeta chimeric antigen receptor and cultured (KTE-X19) for: Treatment of mantle cell lymphoma
Date of first decision: 13/11/2019, Positive, Last updated: 25/01/2021 -
List item
Orphan designation: recombinant human interleukin-3 truncated diphtheria toxin fusion protein for: Treatment of blastic plasmacytoid dendritic cell neoplasm
Date of first decision: 11/11/2015, Positive, Last updated: 25/01/2021 -
List item
Orphan designation: Fenfluramine hydrochloride for: Treatment of Dravet syndrome
Date of first decision: 18/12/2013, Positive, Last updated: 08/01/2021 -
List item
Orphan designation: Autologous CD34+ cells transfected with lentiviral vector containing the human arylsulfatase A cDNA for: treatment of metachromatic leukodystrophy
Date of first decision: 13/04/2007, Positive, Last updated: 22/12/2020 -
List item
Orphan designation: Adeno-associated viral vector serotype 9 containing the human SMN gene (onasemnogene abeparvovec) for: Treatment of spinal muscular atrophy
Date of first decision: 19/06/2015, Positive, Last updated: 10/12/2020 -
List item
Orphan designation: (S)-2-nitro-6-(4-trifluoromethoxy)benzyloxy)-6,7-dihydro-5H-imidazo[2,1-b] [1,3] oxazine (pretomanid) for: Treatment of tuberculosis
Date of first decision: 29/11/2007, Positive, Last updated: 09/12/2020 -
List item
Orphan designation: Obiltoxaximab for: Treatment of anthrax
Date of first decision: 24/08/2018, Positive, Last updated: 27/11/2020 -
List item
Orphan designation: Synthetic double-stranded siRNA oligonucleotide directed against hydroxyacid oxidase 1 mRNA and covalently linked to a ligand containing three N-acetylgalactosamine residues (lumasiran) for: Treatment of primary hyperoxaluria
Date of first decision: 21/03/2016, Positive, Last updated: 25/11/2020 -
List item
Orphan designation: Humanised monoclonal antibody against P-selectin (crizanlizumab) for: Treatment of sickle cell disease
Date of first decision: 09/08/2012, Positive, Last updated: 24/11/2020 -
List item
Orphan designation: Recombinant human beta-glucuronidase (vestronidase alfa) for: Treatment of mucopolysaccharidosis type VII (Sly syndrome)
Date of first decision: 21/03/2012, Positive, Last updated: 11/11/2020 -
List item
Orphan designation: (3S)-3-{4-[7-(aminocarbonyl)-2H-indazol-2-yl] phenyl} piperidine tosilate monohydrate salt (niraparib) for: Treatment of ovarian cancer
Date of first decision: 04/08/2010, Positive, Last updated: 06/11/2020